CyNCh is a multi-center, placebo-controlled clinical trial of children ages 8 to 17 years with biopsy-confirmed moderate to severe nonalcoholic fatty liver disease (NAFLD). The primary objective is to evaluate whether 52 weeks of treatment with cysteamine bitartrate delayed-release capsules will result in improvement in liver disease severity.

Centrally scored and masked assessment of histologic improvement in NAFLD between the baseline liver biopsy and follow-up biopsy after 52 weeks of treatment, where improvement is defined as: (1) decrease in NAS of 2 or more and (2) no worsening of fibrosis.

Liver biopsy obtained within 90 days of screening visit and not more than 120 days before randomization

Clinical history consistent with NAFLD

Definite NAFLD based upon liver histology

No evidence of any other liver disease by clinical history or histological evaluation

A histological severity of: NAFLD Activity Score (NAS) ≥ 4.

Sexually active female participants of childbearing potential (i.e., not surgically sterile [defined as tubal ligation, hysterectomy, or bilateral oophorectomy]) must agree to utilize the same two acceptable forms of contraception from screening through completion of the study and to complete a serum pregnancy test at each study visit. The acceptable forms of contraception for this study include hormonal contraceptives (oral, implant, transdermal patch, or injection) at a stable dose for at least 3 months prior to screening, and barrier (condom with spermicide, diaphragm with spermicide). Sexual activity will be ascertained at each study visit for post-menarchal females and if sexually active, subject must verify use of the same 2 acceptable forms of contraception. For pre-pubescent children, a documented attestation of abstinence from their parent or guardian will be acceptable.

Participants must be able to swallow DR Cysteamine tablets with the tablet intact

Written informed consent from parent or legal guardian

Written informed assent from the child

Exclusion Criteria:

There will be no exclusion criteria based on race, ethnicity or gender.

Participants with a current history of the following conditions or any other health issues that make it unsafe for them to participate in the opinion of the Investigators:

Inflammatory bowel disease (if currently active) or prior resection of small intestine

Children who are currently enrolled in a clinical trial or who received an investigational study drug within 180 days of screening or liver biopsy.

Subjects who are not able or willing to comply with the protocol or have any other condition that would impede compliance or hinder completion of the study, in the opinion of the investigator.

Failure to give informed consent

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01529268

Locations

United States, California

University of California, San Diego

San Diego, California, United States, 92103

University of California, San Francisco

San Francisco, California, United States, 94143

United States, Georgia

Emory University

Atlanta, Georgia, United States, 30322

United States, Illinois

Ann & Robert H. Lurie Children's Hospital of Chicago (NWU)

Chicago, Illinois, United States, 60611-2605

United States, Indiana

Indiana University

Indianapolis, Indiana, United States, 46202

United States, Missouri

St. Louis University

St. Louis, Missouri, United States, 63104

United States, New York

Columbia University

New York, New York, United States, 10032

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229-3039

United States, Texas

Texas Children's Hospital

Houston, Texas, United States, 77030

United States, Washington

University of Washington, Seattle Children's Hospital

Seattle, Washington, United States, 98105

Sponsors and Collaborators

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Investigators

Study Director:

Edward Doo, MD

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)